Status:
COMPLETED
Safety and Pharmacokinetics of AT-007 in Healthy Subjects and in Adult Subjects With Classic Galactosemia
Lead Sponsor:
Applied Therapeutics, Inc.
Conditions:
Classic Galactosemia
Eligibility:
All Genders
18-55 years
Phase:
PHASE1
PHASE2
Brief Summary
This study is a first-in-human, randomized, placebo-controlled, 4-Part, single ascending dose (SAD) and multiple ascending dose (MAD) study in healthy adult subjects and adult subjects with Classic Ga...
Detailed Description
The study is designed to assess the safety and PK of AT-007 in healthy subjects and subjects with Classic Galactosemia as well as the effect of AT-007 on biomarkers of galactose metabolism (galactose,...
Eligibility Criteria
Inclusion
- Diagnosis of Classic Galactosemia confirmed by evidence of absent or significantly decreased (\<1%) GALT activity in red blood cells and by GALT gene analysis
- Urine galactitol \>100 mmol/mol creatinine
- Galactose-restricted diet
Exclusion
- Complications of CG resulting in disability that, in the opinion of the Investigator, may prevent the subject from completing all study requirements (e.g., severe neurological deficits, severe cognitive impairment, or severe language difficulty).
- Renal disease (eGFR \< 90 mL/min/1.73 m2 or albuminuria).
Key Trial Info
Start Date :
June 21 2019
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 14 2021
Estimated Enrollment :
114 Patients enrolled
Trial Details
Trial ID
NCT04117711
Start Date
June 21 2019
End Date
December 14 2021
Last Update
May 30 2024
Active Locations (4)
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1
Anaheim Clinical Trials, LLC
Anaheim, California, United States, 92801
2
Atlanta Center for Medical Research
Atlanta, Georgia, United States, 30331
3
Brigham and Women's Hospital
Boston, Massachusetts, United States, 02115
4
ICON Clinical Research
San Antonio, Texas, United States, 78209